Novartis Wraps Zolgensma Price, Availability Negotiations in Canada
Novartis has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding pricing and availability of Zolgensma (onasemnogene abeparvovec), its approved gene therapy for spinal muscular atrophy (SMA), for pediatric patients.
With this negotiation completed, individual public drug plans in Canada will now make final decisions about the coverage and availability of Zolgensma for children with SMA, according to the company.
“We know the SMA community has been waiting for this day and we’re thrilled that children with SMA are one step closer to having provincial access to Zolgensma,” Andrea Marazzi, country pharma organization head at Novartis Pharmaceuticals Canada, said in a press release.
“We look forward to working with provincial jurisdictions to make Zolgensma available as quickly as possible through public formularies to ensure all children who may benefit from this one-time therapy can access it,” Marazzi added.
Leaders from Cure SMA Canada, a nonprofit that provides direct family support for those with SMA in addition to funding Canadian research projects, called the report “encouraging news for the SMA community across Canada.”
“However, motor neuron damage from SMA won’t stop while the provincial reimbursement process continues,” said Susi Vander Wyk, the organization’s executive director. “We hope that provinces move swiftly to add Zolgensma to their drug plans.”
Zolgensma was approved by Health Canada late last year. The approval covered children with three or fewer copies of SMN2, or who have infantile-onset SMA, also known as type 1 disease.
Earlier this month, the Canadian Agency for Drugs and Technologies in Health (CADTH) — an independent, nonprofit organization that provides evidence-based advice to health policymakers in Canada — recommended the gene therapy be reimbursed for infants up to age 6 months.
However, CADTH did not recommend reimbursement of Zolgensma for older patients, citing lack of evidence — a move that drew criticism from rare disease advocates.
The pCPA and Novartis then negotiated to create a letter of intent detailing the terms and conditions for funding Zolgensma. Specific details about the agreement were not disclosed.
The next step will be for individual public plans (e.g., insurance plans in each Canadian province) to reach their own decisions about coverage and reimbursement, at which point people on those plans who are covered would have access to the therapy.
“Access to Zolgensma will offer children with SMA the possibility of disease stabilization, drastically changing the outcome of this disease,” Vander Wyk said.
SMA is caused by mutations in the gene SMN1. Zolgensma is a one-time therapy that uses a modified viral vector to deliver a non-mutated version of this gene to the body’s cells. The therapy has been shown to improve motor function in children with SMA in clinical trials and in real-world data.